Should the FDA Approve Experimental Treatment for Severe Diseases?

I’ve never had the pain and agony of having a kid who is truly sick.  Broken bones and minor surgeries don’t count.  Even one of my kid’s bout with malaria doesn’t rate, as this illness was easily cured.

 

Parents of kids with chronic illnesses would sacrifice anything to help their kids get better or to suffer less.  In the news recently is a conflict between families of kids with Duchenne muscular dystrophy and the Food and Drug Administration (FDA).  A very small study of an experimental drug called eteplirsen suggested some benefit.   Understandably, the families want the FDA to grant approval so that their kids and others could have access to this drug that will fight a dreadful disease that is fatal.  Families argue that these kids have nothing to lose and can’t wait another 5 years waiting for more definite evidence of efficacy to emerge. 

 

The FDA is legally required to approve drugs that are safe and effective. Obviously, the definitions of safe and effective are subjective, but the agency requires that a reasonable threshold be crossed for both of these parameters.  Gray areas create agonizing conundrums for agency officials and patient advocates.